Immunotherapy in neuromuscular disorders

Hamza Malek MD (

Dr. Malek of Canton-Potsdam Hospital has no relevant financial relationships to disclose.

Jinny O Tavee MD (

Dr. Tavee of Northwestern University Feinberg School of Medicine has no relevant financial relationships to disclose.

Emma Ciafaloni MD, editor. (

Dr. Ciafaloni of the University of Rochester received personal compensation for serving on advisory boards and/or as a consultant for Avexis, Biogen, Pfizer, PTC Therapeutics, Sarepta, Ra pharma, Wave, and Strongbridge Biopharma; and for serving on a speaker’s bureau for Biogen. Dr Ciafaloni also received research and/or grant support from Orphazyme, PTC Therapeutics, Santhera, and Sarepta.

Originally released September 8, 2011; last updated February 11, 2020; expires February 11, 2023

This article includes discussion of immunotherapy in neuromuscular disorders, chronic inflammatory polyradiculoneuropathy, multifocal motor neuropathy, paraproteinemic neuropathies, myasthenia gravis, and inflammatory myopathies. The foregoing terms may include synonyms, similar disorders, variations in usage, and abbreviations.


An increasing number of immunotherapies are being used to treat the heterogeneous group of neuromuscular diseases believed to have an autoimmune pathogenesis. Current treatment options are discussed for the most frequent of these diseases: inflammatory neuropathies, myasthenia gravis, and the inflammatory myopathies. New therapies used in these diseases, including biological agents (monoclonal antibodies or recombinant proteins), are also reviewed.

Key points


• Classic immunosuppressants remain the most beneficial and widely used drugs for immune-mediated neuromuscular diseases.


• Most patients with immune-mediated neuromuscular diseases experience improvement of symptoms and quality-of-life measures with appropriate treatment.


• Several specific therapies have emerged as potential treatments for immune-mediated neuromuscular diseases.


• Subcutaneous immunoglobulin is a less expensive and better tolerated option than intravenous immunoglobulin.


• Biological agents have emerged as effective therapies for patients with treatment-resistant immune-mediated neuromuscular diseases.

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